Mount Sinai joins with Paradigm and ReqMed to repurpose drug for treatment of MPS

Reviewed by James Ives, M.Psych. (Editor)Jan 21 2019

The Icahn School of Medicine at Mount Sinai has entered into two exclusive licenses to develop the drug pentosan polysulfate sodium for the treatment of mucopolysaccharidosis (MPS), with Australia-based company Paradigm Biopharmaceuticals Ltd.  and ReqMed Company, Ltd. of Japan.

MPS is a group of 11 lysosomal storage diseases, metabolic disorders caused by genetic defects that affect the skeletal system, skin, heart, brain, and other organ systems and can lead to disability or death. These conditions occur in about 3.5 out of 100,000 births. Calogera Simonaro, PhD, Research Professor in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine, demonstrated the potential use of the drug through several proof-of-concept studies carried out in animal models of MPS. Pentosan polysulfate sodium, normally used as an anti-inflammatory for bladder pain, was repurposed in Dr. Simonaro's lab to improve connective tissue affected by MPS. Current therapies that exist for these diseases, such as enzyme replacement therapy and bone marrow transplantation, are costly, require highly invasive procedures, and can have limited efficacy. These therapies can slow, but often do not halt, progression of the disease.

"By using a drug that is already approved, we should be able to fast-track [pentosan polysulfate sodium] to patients and address an important unmet medical need," said Edward Schuchman, PhD, the Genetic Disease Foundation - Francis Crick Professor of Genetics and Genomic Sciences at the Icahn School of Medicine and the Department's Vice Chair for Research. "Using one drug for several different lysosomal storage diseases changes the approach currently used for treating these diseases." Paradigm will develop the drug in the United States, the European Union, and other territories, and ReqMed will develop the drug in Japan and other select Asian territories. The financial backing and infrastructure provided by these partnerships will facilitate blinded studies for regulatory approval. Drs. Schuchman and Simonaro believe pentosan polysulfate sodium can also be used to treat other lysosomal storage diseases, including Fabry and Gaucher diseases.

"Mount Sinai innovators recognized the value of repurposing an existing therapeutic for a new indication," said Erik Lium, PhD, Executive Vice President of Mount Sinai Innovation Partners. "We look forward to working with Paradigm and ReqMed to advance pentosan polysulfate sodium for the treatment of MPS."